KR102137087B1 - 척수성 근위축증을 치료하기 위한 화합물 - Google Patents

척수성 근위축증을 치료하기 위한 화합물 Download PDF

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KR102137087B1
KR102137087B1 KR1020147025275A KR20147025275A KR102137087B1 KR 102137087 B1 KR102137087 B1 KR 102137087B1 KR 1020147025275 A KR1020147025275 A KR 1020147025275A KR 20147025275 A KR20147025275 A KR 20147025275A KR 102137087 B1 KR102137087 B1 KR 102137087B1
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pyrido
pyrimidin
methyl
pyrazin
alkyl
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KR1020147025275A
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KR20140121482A (ko
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홍얀 퀴
순규 최
아말 다카
개리 미첼 카르프
자나 나라심한
니콜라이 나리쉬킨
안토니 에이 터포프
말라 엘 위트올
엘렌 웰치
매튜 쥐 월
티아늘 양
난징 장
샤오얀 장
신 자오
루크 그린
엠마누엘 피나르드
하산 라트니
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피티씨 테라퓨틱스, 인크.
에프. 호프만-라 로슈 아게
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Priority to KR1020207020788A priority Critical patent/KR102341596B1/ko
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/70Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings condensed with carbocyclic rings or ring systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Pyridine Compounds (AREA)
  • Steroid Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020147025275A 2012-02-10 2013-02-08 척수성 근위축증을 치료하기 위한 화합물 KR102137087B1 (ko)

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KR1020207020788A KR102341596B1 (ko) 2012-02-10 2013-02-08 척수성 근위축증을 치료하기 위한 화합물

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US201261597523P 2012-02-10 2012-02-10
US61/597,523 2012-02-10
PCT/US2013/025292 WO2013119916A2 (en) 2012-02-10 2013-02-08 Compounds for treating spinal muscular atrophy

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KR102137087B1 true KR102137087B1 (ko) 2020-07-24

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KR1020147025275A KR102137087B1 (ko) 2012-02-10 2013-02-08 척수성 근위축증을 치료하기 위한 화합물

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US (6) US9586955B2 (de)
EP (2) EP3406252B1 (de)
JP (2) JP6092897B2 (de)
KR (2) KR102341596B1 (de)
CN (2) CN104349777B (de)
AR (1) AR092794A1 (de)
AU (2) AU2013216870B2 (de)
BR (1) BR112014019750B1 (de)
CA (1) CA2863874C (de)
CL (1) CL2014002100A1 (de)
CO (1) CO7061082A2 (de)
CR (1) CR20140376A (de)
DK (1) DK2812004T3 (de)
EA (2) EA037123B1 (de)
EC (1) ECSP14017269A (de)
ES (1) ES2697174T3 (de)
HK (1) HK1202077A1 (de)
HU (1) HUE039779T2 (de)
IL (2) IL233959A (de)
MA (1) MA35920B1 (de)
MX (2) MX354074B (de)
NZ (1) NZ628186A (de)
PE (1) PE20142364A1 (de)
PH (2) PH12014501786B1 (de)
PL (1) PL2812004T3 (de)
SG (2) SG10201609188WA (de)
TR (1) TR201813877T4 (de)
TW (2) TWI585085B (de)
UA (1) UA116981C2 (de)
WO (1) WO2013119916A2 (de)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200093066A (ko) * 2012-02-10 2020-08-04 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물

Families Citing this family (72)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10327940B2 (en) 2008-11-09 2019-06-25 3D Systems, Inc. Spiral brace
EP2797592B1 (de) 2011-12-30 2019-08-28 PTC Therapeutics, Inc. Verbindungen zur behandlung von spinaler muskelatrophie
EP2809322B9 (de) 2012-01-26 2019-10-30 PTC Therapeutics, Inc. Verbindungen zur behandlung von spinaler muskelatrophie
WO2013130689A1 (en) 2012-03-01 2013-09-06 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014102778A2 (en) * 2012-12-24 2014-07-03 Ramot At Tel-Aviv University Ltd. Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
CA2915764A1 (en) * 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
US10195202B2 (en) 2013-12-19 2019-02-05 Ptc Therapeutics, Inc. Methods for modulating the amount of RNA transcripts
PT3143025T (pt) 2014-05-15 2019-12-03 Hoffmann La Roche Compostos para tratamento da atrofia muscular espinhal
US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
EP3242763A4 (de) * 2015-01-05 2018-08-29 Sikorsky Aircraft Corporation Integrierter schwingungsdämpfer für generativ gefertigte struktur und verfahren
EP3298017B1 (de) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Verbindungen zur behandlung von spinaler muskelatrophie
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CA3000971A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
AU2016353961B2 (en) * 2015-11-12 2019-08-29 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
CA2996657A1 (en) * 2015-11-12 2017-05-18 Kathleen Dorothy MCCARTHY Compounds for treating amyotrophic lateral sclerosis
EP3386978B1 (de) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Verbrückte piperidinderivate
PL3386511T3 (pl) 2015-12-10 2021-11-08 Ptc Therapeutics, Inc. Sposoby leczenia choroby huntingtona
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP7049248B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー
WO2017186668A1 (en) 2016-04-28 2017-11-02 F. Hoffmann-La Roche Ag A process for the preparation of 2-pyrazolo[1,5-a]pyrazin-2-ylpyrido[1,2-a]pyrimidin-4-one
WO2017218884A1 (en) 2016-06-16 2017-12-21 Ionis Pharmaceuticals, Inc. Combinations for the modulation of smn expression
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing
WO2018119374A1 (en) 2016-12-22 2018-06-28 Cadent Therapeutics Nmda receptor modulators and uses thereof
MX2019014514A (es) 2017-06-05 2020-07-20 Ptc Therapeutics Inc Compuestos para tratar la enfermedad de huntington.
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
MX2019015580A (es) * 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
EP3644996B1 (de) * 2017-06-28 2023-07-26 PTC Therapeutics, Inc. Verfahren zur behandlung von morbus huntington
BR122020005073A2 (pt) 2017-08-04 2020-10-13 Skyhawk Therapeutics, Inc. Composto, composição farmacêutica e uso do composto
AU2018322319B2 (en) 2017-08-25 2021-08-05 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP3684766A1 (de) * 2017-09-22 2020-07-29 H. Hoffnabb-La Roche Ag Verfahren zur herstellung von 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-on-derivaten
EA202092001A1 (ru) 2018-03-27 2021-01-29 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения болезни гентингтона
EP3814360A1 (de) 2018-06-27 2021-05-05 PTC Therapeutics, Inc. Heteroarylverbindungen zur behandlung von morbus huntington
KR20210025625A (ko) * 2018-06-27 2021-03-09 리보르나 바이오사이언시스, 아이엔씨. 척수성 근위축증 예방제 또는 치료제
CA3103976A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
CR20210125A (es) 2018-08-03 2021-06-30 Cadent Therapeutics Inc Moduladores del receptor nmda heteroaromático y usos de los mismos
WO2020033473A1 (en) * 2018-08-07 2020-02-13 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
EP3920915A4 (de) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. Verfahren und zusammensetzungen zur modulierung der spleissung
CN113677344A (zh) 2019-02-06 2021-11-19 斯基霍克疗法公司 用于调节剪接的方法和组合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN110540535B (zh) * 2019-10-23 2020-07-31 上海再启生物技术有限公司 适合放大制备4-(6-氨基吡啶-3-基)取代哌啶的工艺方法
CN111116576A (zh) * 2019-12-01 2020-05-08 北京师范大学 一种喹嗪酮类化合物及其制备方法
AU2020394867A1 (en) 2019-12-02 2022-05-26 Storm Therapeutics Limited Polyheterocyclic compounds as METTL3 inhibitors
EP4097092A1 (de) 2020-01-28 2022-12-07 Protego Biopharma, Inc. Verbindungen, zusammensetzungen und verfahren zur stabilisierung von transthyretin und hemmung von transthyretin-missfaltung
AU2021228284A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN115551847A (zh) 2020-02-28 2022-12-30 雷密克斯医疗公司 用于调节核酸剪接的哒嗪衍生物
US20230142338A1 (en) 2020-02-28 2023-05-11 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
JP2023515619A (ja) 2020-02-28 2023-04-13 リミックス セラピューティクス インコーポレイテッド スプライシングを調節するための化合物及び方法
CN115279379A (zh) 2020-02-28 2022-11-01 Ionis 制药公司 用于调节smn2的化合物和方法
WO2021207530A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN115551593A (zh) 2020-04-08 2022-12-30 雷密克斯医疗公司 用于调节剪接的化合物和方法
US20230287410A1 (en) 2020-05-11 2023-09-14 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
IL299543A (en) 2020-07-02 2023-02-01 Remix Therapeutics Inc 2-(Indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as nucleic acid splicing modulators and for the treatment of multiple diseases
JP2023532331A (ja) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての5-[5-(ピペリジン-4-イル)チエノ[3,2-c]ピラゾール-2-イル]インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法
WO2022070071A1 (en) * 2020-09-30 2022-04-07 Janssen Biotech, Inc. Dihydroorotate dehydrogenase inhibitors
JP2024514421A (ja) 2021-03-17 2024-04-02 エフ. ホフマン-ラ ロシュ アーゲー 新規チアゾロピリミジノン誘導体
WO2023009585A2 (en) 2021-07-28 2023-02-02 Protego Biopharma, Inc. Transthyretin stabilizing compounds
WO2023034833A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034827A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034811A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
AU2022340791A1 (en) 2021-08-30 2024-03-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
TW202333689A (zh) * 2021-10-13 2023-09-01 美商雷密克斯醫療公司 調節剪接之化合物及方法
TW202346305A (zh) 2022-01-05 2023-12-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
TW202337442A (zh) 2022-01-05 2023-10-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
CN117263936B (zh) * 2023-11-21 2024-02-23 中国中医科学院医学实验中心 一种咪唑并[1, 2-a]吡啶衍生物及其制备方法和在中枢神经系统渗透性HDAC6抑制药物中的应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011050245A1 (en) * 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3558618A (en) * 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
US4122274A (en) * 1977-05-25 1978-10-24 Bristol-Myers Company 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones
JPS56150091A (en) * 1980-03-28 1981-11-20 Janssen Pharmaceutica Nv 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture
US4342870A (en) * 1980-03-28 1982-08-03 Janssen Pharmaceutica N.V. Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives
US5089633A (en) 1987-04-28 1992-02-18 Georgia Tech Research Corporation Substituted isocoumarins
US5726182A (en) * 1990-05-02 1998-03-10 Abbott Laboratories Quinolizinone type compounds
AU4231293A (en) * 1992-05-13 1993-12-13 E.I. Du Pont De Nemours And Company Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides
JPH11510478A (ja) * 1995-06-06 1999-09-14 アボツト・ラボラトリーズ キノリジノン型化合物
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
AU5991699A (en) * 1998-09-21 2000-04-10 Biochem Pharma Inc. Quinolizinones as integrin inhibitors
ATE312820T1 (de) * 1999-10-28 2005-12-15 Trine Pharmaceuticals Inc Pumpeninhibitoren zur freisetzung von medikamenten
WO2001053266A1 (en) * 2000-01-24 2001-07-26 Kinacia Pty Ltd. Therapeutic morpholino-substituted compounds
CA2665516A1 (en) 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Drug efflux pump inhibitor
CA2469316A1 (en) 2001-12-07 2003-06-19 Vertex Pharmaceuticals, Inc. Pyrimidine-based compounds useful as gsk-3 inhibitors
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
AU2003256755A1 (en) 2002-07-24 2004-02-09 Ptc Therapeutics, Inc. Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations
CN1809563A (zh) 2003-06-20 2006-07-26 希龙公司 吡啶并[1,2-a]嘧啶-4-酮化合物用作抗癌药
MXPA06012829A (es) * 2004-05-04 2007-01-26 Warner Lambert Co Pirido[2,3-d]pirimidin-7-onas sustituidas con pirrolilo y derivados de las mismas como agentes terapeuticos.
WO2006078834A1 (en) 2005-01-21 2006-07-27 Janssen Pharmaceutica N.V. Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
AR059339A1 (es) 2006-02-09 2008-03-26 Chugai Pharmaceutical Co Ltd Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
US8337941B2 (en) 2006-07-27 2012-12-25 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP2138493A1 (de) * 2008-06-26 2009-12-30 Sanofi-Aventis Substituierte Pyrimidon-Derivate
US8986935B2 (en) 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8754220B2 (en) * 2009-11-20 2014-06-17 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide M1 receptor positive allosteric modulators
KR20130088002A (ko) * 2010-01-13 2013-08-07 재단법인 한국파스퇴르연구소 항-감염성 피리도(1,2-a)피리미딘
EP2797592B1 (de) 2011-12-30 2019-08-28 PTC Therapeutics, Inc. Verbindungen zur behandlung von spinaler muskelatrophie
EP2809322B9 (de) 2012-01-26 2019-10-30 PTC Therapeutics, Inc. Verbindungen zur behandlung von spinaler muskelatrophie
SG10201609188WA (en) * 2012-02-10 2016-12-29 Ptc Therapeutics Inc Compounds for treating spinal muscular atrophy
WO2013130689A1 (en) 2012-03-01 2013-09-06 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
PT3143025T (pt) 2014-05-15 2019-12-03 Hoffmann La Roche Compostos para tratamento da atrofia muscular espinhal
EP3298017B1 (de) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Verbindungen zur behandlung von spinaler muskelatrophie

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011050245A1 (en) * 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Jareck et al., Human Molecular Genetics, 2005, 14(14), pp.2003-2018 (2005.6.8.)*
Peng et al., J. Med. Chem., 2011, 54, 7729-7733 (2011.09.29.)*

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200093066A (ko) * 2012-02-10 2020-08-04 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
KR102341596B1 (ko) * 2012-02-10 2021-12-21 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물

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